Using engineered tRNAs to fix genetic mutations causing diseases like cystic fibrosis
In vivo delivery of engineered tRNAs for suppression of nonsense mutations
['FUNDING_R01'] · UNIVERSITY OF ROCHESTER · NIH-10798308
This study is exploring a new way to help people with genetic diseases like cystic fibrosis by using special tools called engineered tRNAs to fix problems in their genes, so their bodies can make the proteins they need to stay healthy.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF ROCHESTER (nih funded) |
| Locations | 1 site (ROCHESTER, UNITED STATES) |
| Trial ID | NIH-10798308 on ClinicalTrials.gov |
What this research studies
This research focuses on addressing genetic diseases caused by nonsense mutations, which lead to the production of incomplete proteins. By delivering engineered tRNAs into the body, the goal is to promote the readthrough of these mutations, allowing for the production of functional proteins. The approach aims to specifically target conditions like cystic fibrosis, where a significant portion of patients have these mutations. The research involves developing and testing a library of modified tRNAs that can effectively bypass the premature stop signals in the genetic code.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with cystic fibrosis who have specific nonsense mutations in the CFTR gene.
Not a fit: Patients without nonsense mutations or those with other forms of cystic fibrosis that do not involve these specific genetic changes may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for patients with genetic disorders caused by nonsense mutations, potentially improving their quality of life.
How similar studies have performed: Previous research has shown promise in using similar approaches to address genetic mutations, indicating potential for success in this novel application.
Where this research is happening
ROCHESTER, UNITED STATES
- UNIVERSITY OF ROCHESTER — ROCHESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LUECK, JOHN D. — UNIVERSITY OF ROCHESTER
- Study coordinator: LUECK, JOHN D.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: bacteria infection, bacterial disease, Bacterial Infections